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Alnylam 5×15

Our “Alnylam 5×15″ and genetic medicine strategy, launched in January 2011, establishes a path for development and commercialization of novel RNAi therapeutics toward genetically defined targets for the treatment of diseases with high unmet medical need.

Products arising from this strategy share several critical characteristics including:

  • the potential to have a major impact in a high unmet need population;
  • a genetically defined, liver-expressed target gene involved in disease and validated in human genetics;
  • the ability to leverage the existing Alnylam RNAi delivery platform;
  • the opportunity to monitor an early, blood-based biomarker with strong disease correlation in Phase 1 clinical trials for human proof of concept; and,
  • the existence of clinically relevant endpoints for the filing of a new drug application (NDA) with a focused patient database and possible accelerated paths for commercialization.

As recently updated in early 2014, we expect to have six to seven RNAi therapeutic programs in clinical development, including two programs in Phase 3, and five to six programs with human proof of concept by the end of 2015.

Development Programs
Discovery
Development
Phase 1
Phase 2
Phase 3
GENETIC MEDICINES
Patisiran (ALN-TTR02)
Revusiran (ALN-TTRsc)
ALN-AT3
ALN-CC5
ALN-AS1
ALN-AAT
ALN-GO1
ALN-TMP
CARDIO-METABOLIC DISEASES
ALN-PCSsc
ALN-AC3
ALN-ANG
ALN-AGT
HEPATIC INFECTIOUS DISEASES
ALN-HBV
ALN-HDV
ALN-PDL
Discovery
Development
Phase 1
Phase 2
Phase 3



GENETIC MEDICINES

TTR-Mediated Amyloidosis
  - Patisiran (ALN-TTR02)
  - Revusiran (ALN-TTRsc)

Hemophilia and Rare Bleeding Disorders - ALN-AT3

Complement-Mediated Diseases - ALN-CC5

Hepatic Porphyrias - ALN-AS1

Alpha-1 Antitrypsin Deficiency - ALN-AAT

Primary Hyperoxaluria - ALN-GO1

Beta-Thalassemia / Iron Overload Disorders - ALN-TMP

Additional Genetic Medicine Programs

CARDIO-METABOLIC DISEASES

Hypercholesterolemia ALN-PCSsc

Mixed Hyperlipidemia and Hypertriglyceridemia - ALN-ANG

Hypertriglyceridemia - ALN-AC3

Hypertension / Preeclampsia - ALN-AGT

Additional Cardio-Metabolic Programs

HEPATIC INFECTIOUS DISEASES

Hepatitis B Virus Infection - ALN-HBV

Hepatitis D Virus Infection - ALN-HDV

Chronic Liver Infection - ALN-PDL

Additional Hepatic ID Programs